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SG11201907056XA - Compositions and methods for the treatment of hemoglobinopathies - Google Patents

Compositions and methods for the treatment of hemoglobinopathies

Info

Publication number
SG11201907056XA
SG11201907056XA SG11201907056XA SG11201907056XA SG11201907056XA SG 11201907056X A SG11201907056X A SG 11201907056XA SG 11201907056X A SG11201907056X A SG 11201907056XA SG 11201907056X A SG11201907056X A SG 11201907056XA SG 11201907056X A SG11201907056X A SG 11201907056XA
Authority
SG
Singapore
Prior art keywords
novartis
international
rule
research
cambridge
Prior art date
Application number
SG11201907056XA
Other languages
English (en)
Inventor
Craig Stephen Mickanin
Christian Schmedt
Jennifer Snead
Susan C Stevenson
Yi Yang
Original Assignee
Novartis Ag
Intellia Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Novartis Ag, Intellia Therapeutics Inc filed Critical Novartis Ag
Publication of SG11201907056XA publication Critical patent/SG11201907056XA/en

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
SG11201907056XA 2017-02-06 2018-02-05 Compositions and methods for the treatment of hemoglobinopathies SG11201907056XA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762455464P 2017-02-06 2017-02-06
PCT/IB2018/050712 WO2018142364A1 (fr) 2017-02-06 2018-02-05 Compositions et méthodes pour le traitement d'hémoglobinoses

Publications (1)

Publication Number Publication Date
SG11201907056XA true SG11201907056XA (en) 2019-08-27

Family

ID=61526837

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11201907056XA SG11201907056XA (en) 2017-02-06 2018-02-05 Compositions and methods for the treatment of hemoglobinopathies

Country Status (18)

Country Link
US (2) US11466271B2 (fr)
EP (1) EP3577223A1 (fr)
JP (3) JP2020505934A (fr)
KR (2) KR20240017098A (fr)
CN (2) CN110546262B (fr)
AR (1) AR110962A1 (fr)
AU (3) AU2018215726B2 (fr)
BR (1) BR112019016205A2 (fr)
CA (1) CA3052275A1 (fr)
EA (1) EA201991862A1 (fr)
IL (2) IL268406B2 (fr)
MX (1) MX2019009361A (fr)
MY (1) MY203407A (fr)
PH (1) PH12019501812A1 (fr)
SA (1) SA519402400B1 (fr)
SG (1) SG11201907056XA (fr)
TW (1) TW201839136A (fr)
WO (1) WO2018142364A1 (fr)

Families Citing this family (57)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10704021B2 (en) 2012-03-15 2020-07-07 Flodesign Sonics, Inc. Acoustic perfusion devices
US20150166982A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting pi3k point mutations
WO2015105955A1 (fr) 2014-01-08 2015-07-16 Flodesign Sonics, Inc. Dispositif d'acoustophorèse avec double chambre acoustophorétique
US11242525B2 (en) 2014-03-26 2022-02-08 Editas Medicine, Inc. CRISPR/CAS-related methods and compositions for treating sickle cell disease
US11708572B2 (en) 2015-04-29 2023-07-25 Flodesign Sonics, Inc. Acoustic cell separation techniques and processes
US11377651B2 (en) 2016-10-19 2022-07-05 Flodesign Sonics, Inc. Cell therapy processes utilizing acoustophoresis
CA2986310A1 (fr) 2015-05-11 2016-11-17 Editas Medicine, Inc. Systemes crispr/cas9 optimises et procedes d'edition de genes dans des cellules souches
CN116334142A (zh) 2015-06-09 2023-06-27 爱迪塔斯医药公司 用于改善移植的crispr/cas相关方法和组合物
AU2016342380B2 (en) 2015-10-23 2022-04-07 President And Fellows Of Harvard College Nucleobase editors and uses thereof
US11214789B2 (en) 2016-05-03 2022-01-04 Flodesign Sonics, Inc. Concentration and washing of particles with acoustics
KR102827276B1 (ko) 2016-08-03 2025-07-01 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
WO2018031683A1 (fr) 2016-08-09 2018-02-15 President And Fellows Of Harvard College Protéines de fusion cas9-recombinase programmables et utilisations associées
WO2018039438A1 (fr) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation d'acides aminés non naturels dans des protéines au moyen de l'édition de bases
JP7588390B2 (ja) 2016-10-14 2024-11-22 プレジデント アンド フェローズ オブ ハーバード カレッジ 核酸塩基エディターのａａｖ送達
WO2018119359A1 (fr) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Édition du gène récepteur ccr5 pour protéger contre l'infection par le vih
TW201839136A (zh) * 2017-02-06 2018-11-01 瑞士商諾華公司 治療血色素異常症之組合物及方法
EP3592853A1 (fr) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression de la douleur par édition de gène
WO2018165631A1 (fr) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Vaccin contre le cancer
US11542496B2 (en) 2017-03-10 2023-01-03 President And Fellows Of Harvard College Cytosine to guanine base editor
EP3596217A1 (fr) 2017-03-14 2020-01-22 Editas Medicine, Inc. Systèmes et méthodes pour le traitement d'hémoglobinopathies
EP3601562A1 (fr) 2017-03-23 2020-02-05 President and Fellows of Harvard College Éditeurs de nucléobase comprenant des protéines de liaison à l'adn programmable par acides nucléiques
WO2018209158A2 (fr) 2017-05-10 2018-11-15 Editas Medicine, Inc. Systèmes et procédés de nucléase guidé par crispr/arn
US11560566B2 (en) 2017-05-12 2023-01-24 President And Fellows Of Harvard College Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation
WO2019003193A1 (fr) * 2017-06-30 2019-01-03 Novartis Ag Méthodes pour le traitement d'une maladie à l'aide de systèmes d'édition de gènes
WO2019014564A1 (fr) 2017-07-14 2019-01-17 Editas Medicine, Inc. Systèmes et procédés d'intégration ciblée et d'édition du génome et détection de celle-ci à l'aide de sites d'amorçage intégrés
CN111801345A (zh) 2017-07-28 2020-10-20 哈佛大学的校长及成员们 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物
WO2019139645A2 (fr) 2017-08-30 2019-07-18 President And Fellows Of Harvard College Éditeurs de bases à haut rendement comprenant une gam
AU2018352592C1 (en) 2017-10-16 2025-09-25 Beam Therapeutics, Inc. Uses of adenosine base editors
EP3704245A1 (fr) 2017-11-01 2020-09-09 Novartis AG Arn synthétiques et procédés d'utilisation
KR102439221B1 (ko) 2017-12-14 2022-09-01 프로디자인 소닉스, 인크. 음향 트랜스듀서 구동기 및 제어기
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
EP3765614A1 (fr) 2018-03-14 2021-01-20 Editas Medicine, Inc. Systèmes et méthodes pour le traitement d'hémoglobinopathies
KR20250134703A (ko) 2018-05-11 2025-09-11 빔 테라퓨틱스, 인크. 프로그래밍가능한 염기 편집기 시스템을 이용하여 병원성 아미노산을 치환하는 방법
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
US12281338B2 (en) 2018-10-29 2025-04-22 The Broad Institute, Inc. Nucleobase editors comprising GeoCas9 and uses thereof
EP3887521A1 (fr) * 2018-11-29 2021-10-06 Editas Medicine, Inc. Systèmes et méthodes pour le traitement d'hémoglobinopathies
WO2020112195A1 (fr) * 2018-11-30 2020-06-04 Yale University Compositions, technologies et procédés d'utilisation de plérixafor pour améliorer l'édition de gènes
KR20210102309A (ko) * 2018-12-05 2021-08-19 프레드 헛친슨 켄서 리서치 센터 유전적으로-변형된 세포를 제작하는 줄어든, 그리고 최소한의 조작
WO2020154500A1 (fr) 2019-01-23 2020-07-30 The Broad Institute, Inc. Protéines chargées supernégativement et utilisations associées
WO2020168133A1 (fr) 2019-02-13 2020-08-20 Beam Therapeutics Inc. Compositions et méthodes de traitement d'hémoglobinopathies
US20230078265A1 (en) 2019-03-19 2023-03-16 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
US20220193142A1 (en) * 2019-04-30 2022-06-23 Edigene Inc. Method for predicting effectiveness of treatment of hemoglobinopathy
CN111939271A (zh) * 2019-04-30 2020-11-17 博雅辑因（北京）生物科技有限公司 一种血红蛋白病治疗有效性预测方法
CN110106203B (zh) * 2019-05-24 2023-08-11 中国医学科学院血液病医院（血液学研究所） 一种新型hbb过表达载体及其设计方法和应用
WO2021072328A1 (fr) 2019-10-10 2021-04-15 The Broad Institute, Inc. Procédés et compositions pour le prime editing d'arn
AU2020380812A1 (en) * 2019-11-06 2022-06-09 Syros Pharmaceuticals, Inc. Compositions and methods for treating sickle cell disease
CN116096429A (zh) * 2020-04-09 2023-05-09 维乎医疗有限公司 Pcsk9的碱基编辑及其用于治疗疾病的使用方法
JP2023525304A (ja) 2020-05-08 2023-06-15 ザ ブロード インスティテュート，インコーポレーテッド 標的二本鎖ヌクレオチド配列の両鎖同時編集のための方法および組成物
CN111575306B (zh) * 2020-06-11 2022-08-05 南方医科大学 一种激活红细胞中γ珠蛋白基因表达的方法
US20230348938A1 (en) * 2020-08-14 2023-11-02 Navan Technologies, Inc. Methods and apparatuses for biomolecule delivery to primary human hematopoietic stem cells using nanostraws
EP4288541A1 (fr) 2021-02-05 2023-12-13 Christiana Care Gene Editing Institute, Inc. Méthodes et compositions de réduction de l'expression et/ou de l'activité génique
EP4482960A1 (fr) * 2022-02-25 2025-01-01 Vor Biopharma Inc. Compositions et procédés pour la modification génétique par réparation dirigée par homologie
WO2024163679A1 (fr) * 2023-02-01 2024-08-08 Prime Medicine, Inc. Compositions d'édition de génome et méthodes de traitement de la fibrose kystique
CN116445402B (zh) * 2023-05-09 2025-04-25 中国科学院生态环境研究中心 红系祖细胞的扩增培养基、扩增培养方法及应用
WO2024254566A2 (fr) * 2023-06-08 2024-12-12 Editas Medicine, Inc. Systèmes et méthodes pour le traitement d'hémoglobinopathies
WO2025128871A2 (fr) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Nanoparticules lipidiques comprenant des molécules d'arn codant destinées à être utilisées dans l'édition de gènes et en tant que vaccins et agents thérapeutiques
WO2025174765A1 (fr) 2024-02-12 2025-08-21 Renagade Therapeutics Management Inc. Nanoparticules lipidiques comprenant des molécules d'arn codant destinées à être utilisées dans l'édition génique et comme vaccins et agents thérapeutiques

Family Cites Families (237)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5800815A (en) 1903-05-05 1998-09-01 Cytel Corporation Antibodies to P-selectin and their uses
US6309639B1 (en) 1991-02-05 2001-10-30 The Board Of Regents Of The University Of Oklahoma Method for inhibiting an inflammatory response using antibodies to P-selectin glycoprotein ligand
US5213796A (en) 1991-05-06 1993-05-25 Dana Farber Cancer Institute Assay for polyomavirus in humans and uses thereof
US5726014A (en) 1991-06-27 1998-03-10 Genelabs Technologies, Inc. Screening assay for the detection of DNA-binding molecules
US5578444A (en) 1991-06-27 1996-11-26 Genelabs Technologies, Inc. Sequence-directed DNA-binding molecules compositions and methods
ES2197150T3 (es) 1992-05-05 2004-01-01 Aeres Biomedical Limited Anticuerpos contra p-selectina y sus utilizaciones.
US7910624B1 (en) 1995-03-03 2011-03-22 The Trustees Of Boston University Compositions for the treatment of blood disorders
EP1736152A3 (fr) 1993-10-29 2009-10-07 The Trustees Of Boston University Compositions physiologiquement stables d'acide butyrique, de sels et de dérivés d'acide butyrique pour traiter les plaies
US5622701A (en) 1994-06-14 1997-04-22 Protein Design Labs, Inc. Cross-reacting monoclonal antibodies specific for E- and P-selectin
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US7013219B2 (en) 1999-01-12 2006-03-14 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics using zinc finger proteins
US6453242B1 (en) 1999-01-12 2002-09-17 Sangamo Biosciences, Inc. Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites
US7070934B2 (en) 1999-01-12 2006-07-04 Sangamo Biosciences, Inc. Ligand-controlled regulation of endogenous gene expression
US20020164575A1 (en) 1999-09-14 2002-11-07 Sangamo Biosciences, Inc., A Delaware Corporation Gene identification
US6780590B2 (en) 1999-09-14 2004-08-24 Sangamo Biosciences, Inc. Gene identification
IL150069A0 (en) 1999-12-06 2002-12-01 Sangamo Biosciences Inc Methods of using randomized libraries of zinc finger proteins for the identification of gene function
IT1320168B1 (it) 2000-03-13 2003-11-18 Univ Ferrara Oligonucleotidi sintetici in grado di indurre il differenziamentoeritroide.
WO2001085765A2 (fr) 2000-05-12 2001-11-15 Merck Patent Gmbh Identification de mfq-110, proteine humaine a doigt de zinc de type c2h2
ATE544473T1 (de) 2000-11-09 2012-02-15 Cold Spring Harbor Lab Chimäre moleküle zur modulation der genexpression
AU2002219992A1 (en) 2000-12-01 2002-06-11 Sangamo Biosciences, Inc. Targeted regulation of gene expression
WO2002086443A2 (fr) 2001-04-18 2002-10-31 Protein Design Labs, Inc Procedes de diagnostic du cancer du poumon, compositions et procedes de criblage de modulateurs du cancer du poumon
EP1435779A4 (fr) 2001-09-24 2005-06-01 Sangamo Biosciences Inc Modulation de cellules souches au moyen de proteines a doigts de zinc (zfp)
AU2003298574B2 (en) 2002-09-05 2008-04-24 California Institute Of Technology Use of chimeric nucleases to stimulate gene targeting
ATE474460T1 (de) 2002-12-13 2010-08-15 Genetix Pharmaceuticals Inc Therapeutische retrovirus-vektoren für gentherapie
US20050025753A1 (en) 2003-04-30 2005-02-03 Wei Han Methods for production of non-disease causing hemoglobin by ex vivo oligonucleotide gene editing of human stem/progenitor cells
WO2005014791A2 (fr) 2003-08-08 2005-02-17 Sangamo Biosciences, Inc. Methodes et compositions permettant un clivage et une recombinaison cibles
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US8409861B2 (en) 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
CN1906308B (zh) 2003-12-19 2011-09-14 诺华疫苗和诊断公司 小干扰rna的细胞转染制剂、相关组合物以及制备和使用方法
US20050244851A1 (en) 2004-01-13 2005-11-03 Affymetrix, Inc. Methods of analysis of alternative splicing in human
US7972854B2 (en) 2004-02-05 2011-07-05 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
CA2885854C (fr) 2004-04-13 2017-02-21 F. Hoffmann-La Roche Ag Anticorps anti-p-selectine
WO2007014181A2 (fr) 2005-07-25 2007-02-01 Johns Hopkins University Modification specifique au site du genome humain utilisant des nucleases en doigt a zinc personnalisees
US20110212096A1 (en) 2006-12-01 2011-09-01 Scott Rollins Anti-p-selectin antibodies and methods of their use and identification
HUE041957T2 (hu) 2006-12-01 2019-06-28 Novartis Ag Anti-P-szelektin antitestek és eljárások azok alkalmazására gyulladásos betegségek kezelésére
US8945565B2 (en) 2006-12-01 2015-02-03 Selexys Pharmaceuticals Corporation Methods of treating inflammatory or thrombotic conditions with anti-P-selectin antibodies
GB0713183D0 (en) 2007-07-06 2007-08-15 King S College London Method
CA2737180C (fr) 2008-09-15 2019-02-19 Children's Medical Center Corporation Modulation de bcl11a pour le traitement d'hemoglobinopathies
PE20100362A1 (es) 2008-10-30 2010-05-27 Irm Llc Derivados de purina que expanden las celulas madre hematopoyeticas
CN102905763B (zh) 2009-12-23 2015-06-17 诺华股份有限公司 脂质、脂质组合物和使用它们的方法
WO2012075027A1 (fr) 2010-11-29 2012-06-07 Mount Sinai School Of Medicine Acides nucléiques peptidiques chimères intégrés (anpci) utilisables en vue de la génération de cellules souches pluripotentes induites
WO2011103215A1 (fr) 2010-02-16 2011-08-25 Mount Sinai School Of Medicine Acides nucléiques peptidiques chimériques intégrés et leur utilisation
SI2590626T1 (sl) 2010-07-06 2016-01-29 Glaxosmithkline Biologicals S.A. Liposomi z lipidi, ki imajo koristno pKa vrednost, za dostavo RNA
WO2012021632A2 (fr) 2010-08-10 2012-02-16 The Johns Hopkins University Génération et utilisation de cellules souches pluripotentes
US20130189351A1 (en) 2010-08-31 2013-07-25 Novartis Ag Lipids suitable for liposomal delivery of protein coding rna
US9127275B2 (en) 2010-12-10 2015-09-08 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of klf-1 and bcl11a genes
EP2678339B1 (fr) 2011-02-18 2016-07-13 Academia Sinica Méthodes et compositions pour le traitement de la bêta-thalassémie et de la drépanocytose
DE202011050611U1 (de) 2011-07-01 2012-10-09 Makita Corporation Anordnung zum Bereitstellen eines Kraftstoff-Luftgemischs für eine Brennkraftmaschine
EP4014966A1 (fr) 2011-07-06 2022-06-22 GlaxoSmithKline Biologicals S.A. Liposomes ayant un rapport n:p utile pour délivrance de molécules d'arn
WO2013019745A1 (fr) 2011-07-29 2013-02-07 Georgia Health Sciences University Procédés et compositions de modification génétique des cellules
US20140271657A1 (en) 2011-10-12 2014-09-18 Children's Medical Center Corporation Combinatorial compositions and methods of treating hemoglobinopathies
US10696944B2 (en) 2011-10-17 2020-06-30 Massachusetts Institute Of Technology Intracellular delivery
US9394547B2 (en) 2012-01-03 2016-07-19 City University Of Hong Kong Method and apparatus for delivery of molecules to cells
US20130178421A1 (en) 2012-01-06 2013-07-11 Alcon Research, Ltd. Interfering rna delivery system and uses thereof
SG11201404241SA (en) 2012-01-27 2014-08-28 Univ Montreal Pyrimido[4,5-b]indole derivatives and use thereof in the expansion of hematopoietic stem cells
BR112014020625A2 (pt) * 2012-02-24 2017-07-04 Hutchinson Fred Cancer Res polinucleotídeo, polipeptídeo, composição, célula e célula tronco editada por genoma
EP3839050A3 (fr) 2012-04-18 2021-09-29 The Board of Trustees of the Leland Stanford Junior University Ciblage génique non disruptif
SG10201809817UA (en) 2012-05-25 2018-12-28 Univ California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
EP3279315A3 (fr) 2012-05-25 2018-02-28 Cellectis Utilisation de pré-t alpha ou d'un variant fonctionnel de celui-ci pour expanser des lymphocytes t déficients en tcr-alpha
US20150017136A1 (en) 2013-07-15 2015-01-15 Cellectis Methods for engineering allogeneic and highly active t cell for immunotherapy
CA2876076A1 (fr) 2012-06-12 2013-12-19 Soren WARMING Procedes et compositions pour la generation d'alleles a inactivation conditionnelle
WO2014011901A2 (fr) 2012-07-11 2014-01-16 Sangamo Biosciences, Inc. Procédés et compositions pour la délivrance d'agents biologiques
DK3494997T3 (da) 2012-07-25 2019-12-02 Broad Inst Inc Inducerbare dna-bindende proteiner og værktøjer til genomperturbation samt anvendelser deraf
KR102474010B1 (ko) 2012-08-29 2022-12-02 상가모 테라퓨틱스, 인코포레이티드 유전적 병태를 치료하기 위한 방법 및 조성물
ES2714523T3 (es) 2012-09-04 2019-05-28 Cellectis Receptor quimérico de antígenos multicatenario y usos del mismo
CN110669746B (zh) 2012-10-23 2024-04-16 基因工具股份有限公司 用于切割靶dna的组合物及其用途
CN104955943B (zh) 2012-11-27 2018-09-25 儿童医疗中心有限公司 用于胎儿血红蛋白再诱导的靶向bcl11a远端调控元件
CA2977152C (fr) 2012-12-06 2019-04-09 Sigma-Aldrich Co. Llc Modification et regulation du genome basees sur crispr
WO2014093694A1 (fr) 2012-12-12 2014-06-19 The Broad Institute, Inc. Systèmes, procédés et compositions de crispr-nickase cas pour la manipulation de séquences dans les eucaryotes
EP2931899A1 (fr) 2012-12-12 2015-10-21 The Broad Institute, Inc. Génomique fonctionnelle employant des systèmes crispr-cas, des compositions, des procédés, des banques d'inactivation et leurs applications
PL2896697T3 (pl) 2012-12-12 2016-01-29 Broad Inst Inc Projektowanie systemów, sposoby i optymalizowane kompozycje kierujące do manipulacji sekwencją
CN105209621B (zh) 2012-12-12 2021-05-25 布罗德研究所有限公司 对用于序列操纵的改进的系统、方法和酶组合物进行的工程化和优化
WO2014093709A1 (fr) 2012-12-12 2014-06-19 The Broad Institute, Inc. Procédés, modèles, systèmes et appareil pour identifier des séquences cibles pour les enzymes cas ou des systèmes crispr-cas pour des séquences cibles et transmettre les résultats associés
EP4286403A3 (fr) 2012-12-12 2024-02-14 The Broad Institute Inc. Systèmes de composants crispr-cas, procédés et compositions pour la manipulation de séquence
ES2536353T3 (es) 2012-12-12 2015-05-22 The Broad Institute, Inc. Ingeniería de sistemas, métodos y composiciones de guía optimizadas para manipulación de secuencias
ES2576128T3 (es) 2012-12-12 2016-07-05 The Broad Institute, Inc. Modificación por tecnología genética y optimización de sistemas, métodos y composiciones para la manipulación de secuencias con dominios funcionales
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
SG10201912328UA (en) 2012-12-12 2020-02-27 Broad Inst Inc Delivery, Engineering and Optimization of Systems, Methods and Compositions for Sequence Manipulation and Therapeutic Applications
SG10201912991WA (en) 2012-12-17 2020-03-30 Harvard College Rna-guided human genome engineering
CA2901676C (fr) 2013-02-25 2023-08-22 Sangamo Biosciences, Inc. Methodes et compositions pour ameliorer une disruption genique a mediation nuclease
EP3567104A1 (fr) 2013-03-01 2019-11-13 Regents of the University of Minnesota Correction du gène à base de talen
EP2964608B1 (fr) 2013-03-08 2019-08-28 Novartis AG Lipides et compositions lipidiques pour l'administration de principes actifs
US10612043B2 (en) 2013-03-09 2020-04-07 Agilent Technologies, Inc. Methods of in vivo engineering of large sequences using multiple CRISPR/cas selections of recombineering events
KR20170108172A (ko) 2013-03-14 2017-09-26 카리부 바이오사이언시스 인코포레이티드 핵산-표적화 핵산의 조성물 및 방법
US11332719B2 (en) 2013-03-15 2022-05-17 The Broad Institute, Inc. Recombinant virus and preparations thereof
CN113563476A (zh) 2013-03-15 2021-10-29 通用医疗公司 遗传和表观遗传调节蛋白至特定基因组基因座的rna引导的靶向
US20140273230A1 (en) 2013-03-15 2014-09-18 Sigma-Aldrich Co., Llc Crispr-based genome modification and regulation
CA2902709A1 (fr) 2013-03-15 2014-09-25 Global Blood Therapeutics, Inc. Compositions et procedes pour la modulation de l'hemoglobine (s)
LT2970948T (lt) 2013-03-15 2019-03-25 Glaxosmithkline Biologicals Sa Rna gryninimo būdai
US10760064B2 (en) 2013-03-15 2020-09-01 The General Hospital Corporation RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci
US9234213B2 (en) 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
EP2981612B1 (fr) 2013-04-04 2019-07-03 Trustees of Dartmouth College Compositions et procédés pour l'excision in vivo d'adn proviral de vih-1
US20150056629A1 (en) 2013-04-14 2015-02-26 Katriona Guthrie-Honea Compositions, systems, and methods for detecting a DNA sequence
US20160186208A1 (en) 2013-04-16 2016-06-30 Whitehead Institute For Biomedical Research Methods of Mutating, Modifying or Modulating Nucleic Acid in a Cell or Nonhuman Mammal
CA2911292C (fr) 2013-05-13 2023-01-03 Cellectis Recepteur de l'antigene chimere specifique de cd19 et ses utilisations
WO2014184744A1 (fr) 2013-05-13 2014-11-20 Cellectis Procédés de production, par génie génétique, d'un lymphocyte t hautement actif à vocation immunothérapeutique
WO2014186585A2 (fr) 2013-05-15 2014-11-20 Sangamo Biosciences, Inc. Procédés et compositions pour le traitement d'une maladie génétique
EP3004352B1 (fr) 2013-05-24 2017-09-27 Roche Innovation Center Copenhagen A/S Modulateurs oligonucléotidiques de la leucémie lymphocytaire chronique à cellules b/lymphome 11a (bcl11a) et utilisations de ceux-ci
WO2014191521A2 (fr) 2013-05-29 2014-12-04 Cellectis Nouvel échafaudage compact de cas9 dans le système crispr de type ii
AU2014273490B2 (en) 2013-05-29 2019-05-09 Cellectis Methods for engineering T cells for immunotherapy by using RNA-guided Cas nuclease system
EP3603679B1 (fr) 2013-06-04 2022-08-10 President and Fellows of Harvard College Régulation transcriptionnelle guidée par arn
US20140356956A1 (en) 2013-06-04 2014-12-04 President And Fellows Of Harvard College RNA-Guided Transcriptional Regulation
KR102551324B1 (ko) 2013-06-05 2023-07-05 듀크 유니버시티 Rna-가이드 유전자 편집 및 유전자 조절
JP6525971B2 (ja) 2013-06-11 2019-06-05 タカラ バイオ ユーエスエー， インコーポレイテッド タンパク質富化微細小胞、タンパク質富化微細小胞の作製方法及び使用方法
US20150315252A1 (en) 2013-06-11 2015-11-05 Clontech Laboratories, Inc. Protein enriched microvesicles and methods of making and using the same
JP6738728B2 (ja) 2013-06-17 2020-08-19 ザ・ブロード・インスティテュート・インコーポレイテッド 肝臓ターゲティングおよび治療のためのＣＲＩＳＰＲ−Ｃａｓ系、ベクターおよび組成物の送達および使用
EP3011033B1 (fr) 2013-06-17 2020-02-19 The Broad Institute, Inc. Génomique fonctionnelle utilisant des systèmes crispr-cas, procédés de composition, cribles et applications de ces derniers
CN105492611A (zh) 2013-06-17 2016-04-13 布罗德研究所有限公司 用于序列操纵的优化的crispr-cas双切口酶系统、方法以及组合物
CA2915845A1 (fr) 2013-06-17 2014-12-24 The Broad Institute, Inc. Delivrance, modification et optimisation de systemes, procedes et compositions pour cibler et modeliser des maladies et des troubles lies aux cellules post-mitotiques
JP6625971B2 (ja) 2013-06-17 2019-12-25 ザ・ブロード・インスティテュート・インコーポレイテッド 配列操作のためのタンデムガイド系、方法および組成物の送達、エンジニアリングおよび最適化
EP3011035B1 (fr) 2013-06-17 2020-05-13 The Broad Institute, Inc. Test pour l'évaluation quantitative du clivage de sites cibles par une ou plusieurs séquences guides du système crispr-cas
CN105793425B (zh) 2013-06-17 2021-10-26 布罗德研究所有限公司 使用病毒组分靶向障碍和疾病的crispr-cas系统和组合物的递送、用途和治疗应用
CN104239331B (zh) 2013-06-19 2018-10-09 阿里巴巴集团控股有限公司 一种用于实现评论搜索引擎排序的方法和装置
US10011850B2 (en) 2013-06-21 2018-07-03 The General Hospital Corporation Using RNA-guided FokI Nucleases (RFNs) to increase specificity for RNA-Guided Genome Editing
EP3019595A4 (fr) 2013-07-09 2016-11-30 Utilisations thérapeutiques d'édition du génome avec des systèmes crispr/cas
CN105517579B (zh) 2013-07-10 2019-11-15 哈佛大学校长及研究员协会 用于RNA向导的基因调节和编辑的正交Cas9蛋白
DK3019619T3 (da) 2013-07-11 2021-10-11 Modernatx Inc Sammensætninger, der omfatter syntetiske polynukleotider, som koder for crispr-beslægtede proteiner, og syntetiske sgrna'er, og anvendelsesfremgangsmåder
WO2015021426A1 (fr) 2013-08-09 2015-02-12 Sage Labs, Inc. Nouvelle protéine de fusion à base de système crispr/cas et son application en édition de génome
CN105848793B (zh) 2013-08-16 2019-03-05 麻省理工学院 材料选择性的递送至细胞
CN106133138A (zh) 2013-08-22 2016-11-16 先锋国际良种公司 使用引导多核苷酸/cas内切核酸酶系统的基因组修饰及其使用方法
GB201315321D0 (en) 2013-08-28 2013-10-09 Koninklijke Nederlandse Akademie Van Wetenschappen Transduction Buffer
WO2015034872A2 (fr) 2013-09-05 2015-03-12 Massachusetts Institute Of Technology Réglage de populations microbiennes à l'aide de nucléases programmables
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US20150079680A1 (en) 2013-09-18 2015-03-19 Kymab Limited Methods, cells & organisms
US20160237455A1 (en) 2013-09-27 2016-08-18 Editas Medicine, Inc. Crispr-related methods and compositions
US10822606B2 (en) 2013-09-27 2020-11-03 The Regents Of The University Of California Optimized small guide RNAs and methods of use
JP5774657B2 (ja) 2013-10-04 2015-09-09 国立大学法人京都大学 エレクトロポレーションを利用した哺乳類の遺伝子改変方法
WO2015052231A2 (fr) 2013-10-08 2015-04-16 Technical University Of Denmark Système d'édition multiplex
WO2015054507A1 (fr) 2013-10-10 2015-04-16 Pronutria, Inc. Systèmes de production de polypeptides nutritifs et procédés de production et d'utilisation de ceux-ci
WO2015065964A1 (fr) 2013-10-28 2015-05-07 The Broad Institute Inc. Génomique fonctionnelle utilisant des systèmes crispr-cas, compositions, procédés, cribles et applications de ces systèmes
US9850497B2 (en) 2013-11-04 2017-12-26 Regents Of The University Of Minnesota Gene targeting methods and tools
JP2016536021A (ja) 2013-11-07 2016-11-24 エディタス・メディシン，インコーポレイテッド ＣＲＩＳＰＲ関連方法および支配ｇＲＮＡのある組成物
PT3492593T (pt) 2013-11-13 2021-10-18 Childrens Medical Center Regulação da expressão de genes mediada por nucleases
EP3071695A2 (fr) 2013-11-18 2016-09-28 Crispr Therapeutics AG Système crips-cas, matériels et procédés
MX378635B (es) 2013-11-22 2025-03-10 Cellectis Método para diseñar células t resistentes a fármacos de quimioterapia para inmunoterapia.
CN106103699B (zh) 2013-11-28 2019-11-26 地平线探索有限公司 体细胞单倍体人类细胞系
BR112016013207A2 (pt) 2013-12-12 2017-09-26 Massachusetts Inst Technology administração, uso e aplicações terapêuticas dos sistemas crispr-cas e composições para o hbv e distúrbios e doenças virais
MX374529B (es) 2013-12-12 2025-03-06 Broad Inst Inc Suministro, uso y aplicaciones terapeuticas de sistemas y composiciones crispr-cas para edicion del genoma.
WO2015089473A1 (fr) 2013-12-12 2015-06-18 The Broad Institute Inc. Ingénierie de systèmes, procédés et compositions guides optimisées avec de nouvelles architectures pour la manipulation de séquences
EP3470089A1 (fr) 2013-12-12 2019-04-17 The Broad Institute Inc. Administration, utilisation et applications thérapeutiques des systèmes et compositions crispr-cas pour cibler les troubles et les maladies au moyen de composants d'administration de particules
US9994831B2 (en) 2013-12-12 2018-06-12 The Regents Of The University Of California Methods and compositions for modifying a single stranded target nucleic acid
US20150166982A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting pi3k point mutations
EP3080271B1 (fr) 2013-12-12 2020-02-12 The Broad Institute, Inc. Systèmes, procédés et compositions pour manipulation de séquences avec systèmes crispr-cas fonctionnels optimisés
JP6712948B2 (ja) 2013-12-12 2020-06-24 ザ・ブロード・インスティテュート・インコーポレイテッド 組成物、及びヌクレオチドリピート障害におけるｃｒｉｓｐｒ−ｃａｓ系の使用方法
WO2015089364A1 (fr) 2013-12-12 2015-06-18 The Broad Institute Inc. Structure cristalline d'un système crispr-cas, et ses utilisations
AU2014361834B2 (en) 2013-12-12 2020-10-22 Massachusetts Institute Of Technology CRISPR-Cas systems and methods for altering expression of gene products, structural information and inducible modular Cas enzymes
US20150191744A1 (en) 2013-12-17 2015-07-09 University Of Massachusetts Cas9 effector-mediated regulation of transcription, differentiation and gene editing/labeling
EP3872066A1 (fr) 2013-12-19 2021-09-01 Novartis AG Lipides et compositions lipidiques pour l'administration d'agents actifs
ES2908827T3 (es) 2013-12-19 2022-05-04 Novartis Ag Lípidos y composiciones lipídicas para el suministro de agentes activos
US9476065B2 (en) 2013-12-19 2016-10-25 Amyris, Inc. Methods for genomic integration
JP6721508B2 (ja) 2013-12-26 2020-07-15 ザ ジェネラル ホスピタル コーポレイション 多重ガイドｒｎａ
WO2015103153A1 (fr) 2013-12-31 2015-07-09 The Regents Of The University Of California Cristaux de cas9 et procédés d'utilisation correspondants
CA2936312A1 (fr) 2014-01-08 2015-07-16 President And Fellows Of Harvard College Activateurs de genes guides par l'arn
JP2017503514A (ja) 2014-01-24 2017-02-02 ノースカロライナ ステート ユニバーシティーＮｏｒｔｈ Ｃａｒｏｌｉｎａ Ｓｔａｔｅ Ｕｎｉｖｅｒｓｉｔｙ Ｃａｓ９ターゲッティングをガイドする配列に関する方法および組成物
ES2796087T3 (es) 2014-02-03 2020-11-25 Sangamo Therapeutics Inc Métodos y composiciones para el tratamiento de beta talasemia
US10287590B2 (en) 2014-02-12 2019-05-14 Dna2.0, Inc. Methods for generating libraries with co-varying regions of polynuleotides for genome modification
CN113265394B (zh) 2014-02-13 2024-08-06 宝生物工程（美国)有限公司 从核酸的初始集合中耗尽靶分子的方法、以及用于实践其的组合物和试剂盒
WO2015126927A2 (fr) 2014-02-18 2015-08-27 Duke University Compositions destinées à l'inactivation de la réplication d'un virus et leurs procédés de fabrication et d'utilisation
US11186843B2 (en) 2014-02-27 2021-11-30 Monsanto Technology Llc Compositions and methods for site directed genomic modification
WO2015134812A1 (fr) 2014-03-05 2015-09-11 Editas Medicine, Inc. Méthodes et compositions liées à crispr/cas et destinées à traiter le syndrome de usher et la rétinite pigmentaire
EP3116997B1 (fr) 2014-03-10 2019-05-15 Editas Medicine, Inc. Méthodes et compositions associées aux crispr/cas, utilisées dans le traitement de l'amaurose congénitale de leber 10 (lca10)
WO2015139139A1 (fr) 2014-03-20 2015-09-24 UNIVERSITé LAVAL Méthodes crispr et produits pouvant augmenter les taux de frataxine et leurs utilisations
US10451633B2 (en) 2014-03-24 2019-10-22 IMMCO Diagnostics, Inc. Anti-nuclear antibody detection and diagnostics for systemic and non-systemic autoimmune disorders
WO2015148670A1 (fr) 2014-03-25 2015-10-01 Editas Medicine Inc. Procédés et compositions liés à crispr/cas pour traiter une infection par le vih et le sida
WO2015148860A1 (fr) 2014-03-26 2015-10-01 Editas Medicine, Inc. Méthodes et compositions liées à crispr/cas pour traiter la bêta-thalassémie
US11242525B2 (en) 2014-03-26 2022-02-08 Editas Medicine, Inc. CRISPR/CAS-related methods and compositions for treating sickle cell disease
WO2015152305A1 (fr) 2014-03-31 2015-10-08 株式会社サイエンス・ラスター Peptide de croissance pour cellules hématopoïétiques et utilisation dudit peptide de croissance pour cellules hématopoïétiques
WO2015153791A1 (fr) 2014-04-01 2015-10-08 Editas Medicine, Inc. Méthodes et compositions relatives à crispr/cas pour traiter le virus de l'herpès simplex de type 2 (vhs-2)
EP3498845B1 (fr) 2014-04-01 2022-06-22 Editas Medicine, Inc. Procédés et compositions associés à crispr/cas pour le traitement du virus de l'herpès simplex de type 1 (hsv-1)
EP3540061A1 (fr) 2014-04-02 2019-09-18 Editas Medicine, Inc. Méthodes se rapportant à crispr/cas, et compositions pour traiter le glaucome à angle ouvert primaire
CN106170550A (zh) 2014-04-03 2016-11-30 麻省理工学院 用于产生导引rna的方法和组合物
EP3556858A3 (fr) 2014-04-09 2020-01-22 Editas Medicine, Inc. Procédés et compositions associés crispr/cas pour le traitement de la mucoviscidose
HUE051354T2 (hu) 2014-04-14 2021-03-01 Nemesis Bioscience Ltd Terapeutikum
SG10201809157VA (en) 2014-04-18 2018-11-29 Editas Medicine Inc Crispr-cas-related methods, compositions and components for cancer immunotherapy
CN106794260B (zh) 2014-04-25 2021-02-23 儿童医疗中心有限公司 治疗血红蛋白病的组合物和方法
GB201407852D0 (en) 2014-05-02 2014-06-18 Iontas Ltd Preparation of libraries od protein variants expressed in eukaryotic cells and use for selecting binding molecules
WO2015168800A1 (fr) 2014-05-09 2015-11-12 UNIVERSITé LAVAL Prévention et traitement de la maladie d'alzheimer par édition du génome à l'aide du système crispr/cas
US20170088819A1 (en) 2014-05-16 2017-03-30 Vrije Universiteit Brussel Genetic correction of myotonic dystrophy type 1
JP2017517256A (ja) 2014-05-20 2017-06-29 リージェンツ オブ ザ ユニバーシティ オブ ミネソタ 遺伝子配列を編集する方法
US9970001B2 (en) 2014-06-05 2018-05-15 Sangamo Therapeutics, Inc. Methods and compositions for nuclease design
WO2015188109A1 (fr) 2014-06-06 2015-12-10 Regeneron Pharmaceuticals, Inc. Procédés et compositions pour la modification d'un locus ciblé
WO2015191693A2 (fr) 2014-06-10 2015-12-17 Massachusetts Institute Of Technology Procédé d'édition génique
WO2015191911A2 (fr) 2014-06-12 2015-12-17 Clontech Laboratories, Inc. Microvésicules enrichies en protéines et leurs procédés de fabrication et d'utilisation
EP3708663A1 (fr) 2014-06-23 2020-09-16 Regeneron Pharmaceuticals, Inc. Assemblage d'adn mediée par nuclease
WO2015200555A2 (fr) 2014-06-25 2015-12-30 Caribou Biosciences, Inc. Modification d'arn visant à manipuler par génie génétique l'activité de cas9
US20150376587A1 (en) 2014-06-25 2015-12-31 Caribou Biosciences, Inc. RNA Modification to Engineer Cas9 Activity
RU2711740C2 (ru) 2014-06-26 2020-01-21 Регенерон Фармасьютикалз, Инк. Способы и композиции для нацеленных генетических модификаций и способы их применения
US10017770B2 (en) 2014-07-03 2018-07-10 Ut-Battelle, Llc TNT cloning system
WO2016007948A1 (fr) 2014-07-11 2016-01-14 Pioneer Hi-Bred International, Inc. Modification de caractère agronomique au moyen d'arn guide/endonucléase cas, systèmes et procédés d'utilisation
EP3169776B1 (fr) 2014-07-14 2025-09-03 The Regents of The University of California Modulation transcriptionnelle par crispr/cas
US10975406B2 (en) 2014-07-18 2021-04-13 Massachusetts Institute Of Technology Directed endonucleases for repeatable nucleic acid cleavage
US10077453B2 (en) 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
MX382337B (es) 2014-09-04 2025-03-13 Memorial Sloan Kettering Cancer Center Terapia génica de globina y uso de la misma para el tratamiento de hemoglobinopatias.
EP3194570B1 (fr) 2014-09-16 2021-06-30 Sangamo Therapeutics, Inc. Procédés et compositions pour ingénierie génomique à médiation par des nucléases et correction du génome dans des cellules souches hématopoïétiques
WO2016049258A2 (fr) 2014-09-25 2016-03-31 The Broad Institute Inc. Criblage fonctionnel avec systèmes crisp-cas fonctionnels optimisés
WO2016057961A1 (fr) 2014-10-10 2016-04-14 Editas Medicine, Inc. Compositions et procédés pour activer une réparation dirigée par homologie
AU2015342749B2 (en) 2014-11-07 2022-01-27 Editas Medicine, Inc. Methods for improving CRISPR/Cas-mediated genome-editing
EP3230452B1 (fr) 2014-12-12 2025-06-11 The Broad Institute, Inc. Guides désactivés pour facteurs de transcription crispr
WO2016094880A1 (fr) 2014-12-12 2016-06-16 The Broad Institute Inc. Administration, utilisation et applications thérapeutiques de systèmes crispr et compositions pour l'édition de génome de cellules souches hématopoïétiques (hsc)
AU2015370435A1 (en) * 2014-12-24 2017-06-15 Dana-Farber Cancer Institute, Inc. Systems and methods for genome modification and regulation
EP3237615B2 (fr) 2014-12-24 2023-07-26 The Broad Institute, Inc. Crispr présentant ou associé avec un domaine de déstabilisation
WO2016115326A1 (fr) 2015-01-15 2016-07-21 The Board Of Trustees Of The Leland Stanford Junior University Méthodes pour moduler l'édition génomique
EP3247808B1 (fr) 2015-01-21 2021-05-05 Fred Hutchinson Cancer Research Center Plate-forme point-of-care et/ou portative pour thérapie génique
US20180002379A1 (en) 2015-01-21 2018-01-04 Sangamo Therapeutics, Inc. Methods and compositions for identification of highly specific nucleases
AU2016209313B2 (en) 2015-01-21 2021-10-14 Cornell University Viral vectors for prophylaxis and therapy of hemoglobinopathies
WO2016130600A2 (fr) 2015-02-09 2016-08-18 Duke University Compositions et procédés pour l'édition de l'épigénome
US10584321B2 (en) 2015-02-13 2020-03-10 University Of Massachusetts Compositions and methods for transient delivery of nucleases
SG11201706766WA (en) 2015-02-23 2017-09-28 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
EP3268044A2 (fr) 2015-03-11 2018-01-17 The Broad Institute Inc. Traitement sélectif de cancer dépendant de prmt5
EP3274454B1 (fr) 2015-03-25 2021-08-25 Editas Medicine, Inc. Procédés, compositions et constituants liés à crispr/cas
WO2016154579A2 (fr) 2015-03-26 2016-09-29 Editas Medicine, Inc. Conversion génique via crispr/cas
WO2016156404A1 (fr) 2015-03-31 2016-10-06 Glycotope Gmbh Vecteurs d'expression eucaryote comprenant des éléments régulateurs des groupes de gènes de globines
KR102648489B1 (ko) 2015-04-06 2024-03-15 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 Crispr/cas-매개 유전자 조절을 위한 화학적으로 변형된 가이드 rna
ES2835861T5 (en) 2015-05-08 2025-02-18 Childrens Medical Ct Corp Targeting bcl11a enhancer functional regions for fetal hemoglobin reinduction
WO2016182893A1 (fr) 2015-05-08 2016-11-17 Teh Broad Institute Inc. Génomique fonctionnelle utilisant des systèmes crispr-cas pour la mutagenèse de saturation d'éléments non codants, compositions, procédés, bibliothèques et leurs applications
CA2986310A1 (fr) 2015-05-11 2016-11-17 Editas Medicine, Inc. Systemes crispr/cas9 optimises et procedes d'edition de genes dans des cellules souches
HK1251011A1 (zh) 2015-05-12 2019-01-18 Sangamo Therapeutics, Inc. 核酸酶介导的基因表达调控
WO2016201272A1 (fr) 2015-06-12 2016-12-15 King Abdulaziz City For Science And Technology Procédé de diagnostic de patients présentant des troubles provoqués par des mutations mendéliennes
EP3310932B1 (fr) 2015-06-17 2023-08-30 The UAB Research Foundation Complexe crispr/cas9 pour l'édition génomique
US9957501B2 (en) 2015-06-18 2018-05-01 Sangamo Therapeutics, Inc. Nuclease-mediated regulation of gene expression
WO2017053729A1 (fr) 2015-09-25 2017-03-30 The Board Of Trustees Of The Leland Stanford Junior University Édition du génome à médiation par une nucléase de cellules primaires et leur enrichissement
WO2017059241A1 (fr) 2015-10-02 2017-04-06 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Système d'administration de protéine lentivirale pour l'édition génomique guidée par l'arn
US12043843B2 (en) 2015-11-04 2024-07-23 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
EA201891532A1 (ru) 2015-12-28 2019-01-31 Новартис Аг Композиции и способы лечения гемоглобинопатий
AU2017235333B2 (en) * 2016-03-14 2023-08-24 Editas Medicine, Inc. CRISPR/CAS-related methods and compositions for treating beta hemoglobinopathies
SG10202010261YA (en) 2016-04-18 2020-11-27 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
WO2017191503A1 (fr) 2016-05-05 2017-11-09 Crispr Therapeutics Ag Substances et méthodes pour le traitement d'hémoglobinopathies
US20190345450A1 (en) 2016-06-17 2019-11-14 Fred Hutchinson Cancer Research Center Strategies to assess and/or produce cell populations with predictive engraftment potential
WO2018009506A1 (fr) 2016-07-07 2018-01-11 St. Jude Children's Research Hospital Promoteur d'érythrocytes.
WO2018022619A1 (fr) 2016-07-25 2018-02-01 Bluebird Bio, Inc. Variants d'endonucléase de homing bcl11a, compositions et procédés d'utilisation
EP3500271A4 (fr) 2016-08-19 2020-05-13 Bluebird Bio, Inc. Activateurs d'édition du génome
HRP20212025T1 (hr) 2016-08-24 2022-04-01 Sangamo Therapeutics, Inc. Regulacija ekspresije gena pomoću projektiranih nukleaza
SG10201913948PA (en) 2016-08-24 2020-03-30 Sangamo Therapeutics Inc Engineered target specific nucleases
EP3519577A1 (fr) 2016-09-28 2019-08-07 Novartis AG Système de distribution de macromolécules à base de membrane poreuse
TW201839136A (zh) * 2017-02-06 2018-11-01 瑞士商諾華公司 治療血色素異常症之組合物及方法

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